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PTC Therapeutics Receives Buy Ratings Following FDA Approval of Gene Therapy Kebilidi

• PTC Therapeutics received a Buy rating from William Blair analyst Sami Corwin due to the FDA approval of Kebilidi, a gene therapy for AADC deficiency. • Kebilidi is the first gene therapy administered directly to the brain, holding a broad label for treating a wide range of patients with AADC deficiency. • The company's proactive commercial launch preparations, coupled with a priority review voucher, provide financial advantages and growth prospects. • Robert W. Baird also assigned a Buy rating to PTC Therapeutics stock, with a price target of $48.00, reinforcing positive market sentiment.

PTC Therapeutics (PTCT) has garnered positive market sentiment, receiving Buy ratings from analysts following the FDA approval of Kebilidi, a groundbreaking gene therapy for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency. The approval marks a significant milestone as Kebilidi is the first gene therapy to be directly administered to the brain, offering a new treatment option for a wide range of patients affected by this rare genetic disorder.

Analyst Optimism

William Blair analyst Sami Corwin has maintained a bullish stance on PTC Therapeutics, citing the FDA approval of Kebilidi as a key factor. The broad label granted to the therapy allows for the treatment of a diverse patient population, enhancing the company's market presence. Corwin also highlighted the company's proactive approach to the commercial launch, including the identification of centers of excellence and the training of surgeons for the administration procedure.

Financial Advantages

The priority review voucher awarded to PTC Therapeutics adds a layer of financial advantage, as it can be monetized for nondilutive capital. This voucher, combined with promising clinical trial results demonstrating the long-term durability of Kebilidi's effects, further solidifies the company's growth prospects.

Additional Buy Rating

In a separate report, Robert W. Baird also assigned a Buy rating to PTC Therapeutics stock, setting a price target of $48.00. This additional endorsement reinforces the positive outlook for the company following the FDA approval of Kebilidi.

Company Profile

PTC Therapeutics, Inc. is a biopharmaceutical company focused on the discovery and commercialization of medicines for rare diseases and oncology. Founded in 1998 and headquartered in South Plainfield, NJ, the company is dedicated to developing treatments for unmet medical needs.
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FDA Approves PTC Therapeutics' Kebilidi, a Gene Therapy for AADC Deficiency

The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec-tneq), a gene therapy developed by PTC Therapeutics for treating AADC deficiency.
Kebilidi is the first gene therapy approved in the US that is directly administered to the brain, offering a novel approach to treating this rare genetic disorder.

FDA Approves PTC Therapeutics' Kebilidi, First Gene Therapy for AADC Deficiency

The FDA has approved Kebilidi (eladocagene exuparvovec-tneq) from PTC Therapeutics, the first gene therapy for Aromatic L-amino acid decarboxylase (AADC) deficiency.
Kebilidi is an adeno-associated virus vector-based gene therapy designed for both adult and pediatric patients suffering from AADC deficiency, administered via neurosurgical infusion.

FDA Approves PTC Therapeutics' Kebilidi, First Gene Therapy Dosed Directly into the Brain

The FDA has approved Kebilidi (eladocagene exuparvovec), a gene therapy by PTC Therapeutics, for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder.
Kebilidi is the first gene therapy approved in the U.S. that involves direct administration into the brain, offering a novel approach to treating AADC deficiency.

FDA Approves PTC Therapeutics' Gene Therapy KEBILIDI for AADC Deficiency

The FDA has granted accelerated approval to PTC Therapeutics' KEBILIDI, a gene therapy for Aromatic L-amino acid decarboxylase (AADC) deficiency, marking the first directly brain-delivered gene therapy in the US.
KEBILIDI addresses the underlying genetic defect in AADC deficiency by delivering a functional DOPA decarboxylase gene directly into the brain to restore dopamine production.

PTC Therapeutics' Gene Therapy Kebilidi Receives FDA Approval for AADC Deficiency

The FDA has granted accelerated approval to PTC Therapeutics' Kebilidi, a gene therapy for Aromatic L-amino acid decarboxylase (AADC) deficiency, marking the first such therapy approved in the U.S.
Kebilidi delivers a functioning DDC gene directly into the brain, addressing the genetic defect underlying AADC deficiency, a severe neurological disorder affecting infants.

FDA Approves KEBILIDI, First Brain-Delivered Gene Therapy for AADC Deficiency

The FDA has approved KEBILIDI (eladocagene exuparvovec-tneq), a gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency, marking the first brain-delivered gene therapy in the U.S.
KEBILIDI, developed by PTC Therapeutics, is a gene replacement therapy administered directly to the brain to restore dopamine production in patients with AADC deficiency.

FDA Approves PTC Therapeutics' KEBILIDI, First Gene Therapy Delivered Directly to the Brain for AADC Deficiency

The FDA has granted accelerated approval to PTC Therapeutics' KEBILIDI, a gene therapy for AADC deficiency, marking the first such therapy delivered directly to the brain.
KEBILIDI works by using an adeno-associated virus to deliver a functional DOPA decarboxylase gene, restoring dopamine production in patients with AADC deficiency.

FDA Approves PTC Therapeutics' Kebilidi for AADC Deficiency

The FDA has granted accelerated approval to PTC Therapeutics' Kebilidi (eladocagene exuparvovec-tneq) for treating AADC deficiency, a rare genetic disorder.
Kebilidi is a gene therapy designed to deliver a functioning DDC gene directly into the putamen to enhance the AADC enzyme and restore dopamine production.

FDA Approves PTC Therapeutics' KEBILIDI™ for AADC Deficiency

The FDA has granted accelerated approval to KEBILIDI™, a gene therapy developed by PTC Therapeutics, for treating AADC deficiency in both children and adults.
KEBILIDI™ is the first gene therapy approved in the U.S. that involves direct administration to the brain, marking a significant advancement in CNS drug delivery.

FDA Approves PTC Therapeutics' KEBILIDI, a Gene Therapy for AADC Deficiency

The FDA has granted accelerated approval to KEBILIDI (eladocagene exuparvovec-tneq), a gene therapy for AADC deficiency, marking the first FDA-approved gene therapy directly administered to the brain.
KEBILIDI is indicated for both children and adults with AADC deficiency, addressing the full spectrum of disease severity and offering a new treatment option for this rare genetic disorder.

Reference News

[1]
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene ...
markets.businessinsider.com · Nov 14, 2024

William Blair analyst Sami Corwin maintains a Buy rating on PTC Therapeutics (PTCT) stock due to the FDA approval of Keb...

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